Genome editing ready to open doors to new treatments

February 19, 2018

“We knew this technology was coming.”

Jennifer A. Doudna, PhD, professor of chemistry, molecular, and cell biology at the University of California, Berkeley, expounded on dramatic changes in genome editing when she presented the Lila and Murray Gruber Memorial Cancer Research Award and Lectureship.

Awarded the honor for her cancer research achievements, Dr. Doudna’s work focuses on understanding and harnessing RNA-mediated control of the genome, including Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Cas9 bacterial adaptive immunity and related systems. CRISPRs are genetic elements containing direct repeats separated by unique spacers, many of which are identical to sequences found in phage and other foreign genetic elements.

“There has been a succession of technologies to change the DNA sequence,” Dr. Doudna said. “However, it has never been possible to turn the technology into robust tools that worked in many people’s hands because of the time it takes to develop it, the expense, etc.”

Dr. Doudna’s recent work has demonstrated the role of CRISPRs in adaptive immunity, showing that small RNAs derived from CRISPRs (crRNAs) are implemented as homing oligos for the targeted interference of foreign DNA. This technology is being adopted in clinical trials for cancer patients by modifying immune cells. Current studies are now underway in the precise editing of DNA in the mouse brain.

Calling this a “truly exciting time” because of the “precise and accurate nature” of the technology, the CRISPR tool opens the doors to other applications beyond clinical settings. This tool is currently being used in trials in animals that provide human health benefits, specifically in making children’s organs donor-suitable for adults, and changing DNA in germline editing of life cells to transmit to future generations. Although this application can have significant impacts on the treatment of chronic diseases, it faces some scrutiny for the ethical questions it raises, she said.

“This gene engineering technology has an important impact on human health, but presents many social and ethical challenges yet to be answered,” she said.